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Effects of HQK-1001 in Patients With Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01601340
Recruitment Status : Terminated
First Posted : May 18, 2012
Last Update Posted : March 18, 2015
Information provided by (Responsible Party):
HemaQuest Pharmaceuticals Inc.

Brief Summary:
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F in subjects with sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Sickle Cell Disorders Hemoglobin S Disease Sickling Disorder Due to Hemoglobin S Drug: HQK-1001 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 77 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
Study Start Date : July 2012
Actual Primary Completion Date : November 2013
Actual Study Completion Date : December 2013

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Active Comparator: HQK-1001 Drug: HQK-1001
HQK-1001 tablets, twice daily for 48 weeks

Placebo Comparator: Placebo Drug: Placebo
Placebo tablets, twice daily for 48 weeks

Primary Outcome Measures :
  1. Change from baseline in % fetal hemoglobin [ Time Frame: Day 1 through Week 48 ]

Secondary Outcome Measures :
  1. Incidence and number of SCD pain crises and SCD-related complications [ Time Frame: Day 1 through Week 52 ]
  2. Subject reported daily pain scale scores and analgesic use [ Time Frame: 7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48 ]
  3. Change in FACIT Fatigue Scale results [ Time Frame: Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48 ]
  4. Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments [ Time Frame: Day 1 through Week 52 ]
  5. HQK-1001 pharmacokinetic parameters [ Time Frame: 1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48 ]
    A subset of subjects (7) will undergo sampling for detailed analysis of pharmacokinetic parameters (AUC, Cmax) with samples taken pre-dose, and 1, 2, 4, 8, and 10 hours after the morning dose at Week 4.

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Males and females between 12 and 60 years of age
  • Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
  • At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
  • Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
  • If subject has been transfused in the 3 months prior to screening, then Hb A level < 20% at screening
  • Baseline Hb F level obtained within 14 days prior to randomization
  • Able to swallow tablets
  • Able and willing to give informed consent and/or assent
  • If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine pregnancy test prior to dosing on Day 1
  • If a subject is a WCBP, she must agree to use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation
  • Sexually active male subjects who have not had a vasectomy must agree to use latex condoms with WCBP partners or ensure that their partner(s) use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation.

Exclusion Criteria:

  • Assigned to a regular transfusion program
  • Use of erythropoiesis stimulating agents within 90 days prior to screening
  • An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
  • More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
  • Pulmonary hypertension requiring therapy
  • ALT or AST > 3x ULN
  • Serum creatinine > 1.5x ULN
  • Serum amylase levels > 1.5x ULN
  • Serum lipase level > 1.5x ULN
  • A serious, concurrent illness that would limit ability to complete or comply with the study requirements
  • An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
  • History of syncope, clinically significant dysrhythmias or resuscitation from sudden death due to SCD-related complication
  • Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
  • History of pancreatitis
  • Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
  • Current abuse of alcohol or drugs
  • Use of another investigational agent within 4 weeks or 5 half-lives, whichever is longer, prior to screening
  • Currently pregnant or breast feeding a child
  • Known infection with HIV-1
  • Infection with hepatitis B or hepatitis C, such that subjects are currently on anti-viral therapy or will be placed on therapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01601340

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Sponsors and Collaborators
HemaQuest Pharmaceuticals Inc.
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Study Director: Richard Ghalie, MD, MBA HemaQuest Pharmaceuticals Inc.
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: HemaQuest Pharmaceuticals Inc. Identifier: NCT01601340    
Other Study ID Numbers: HQP 1001-SCD-007
First Posted: May 18, 2012    Key Record Dates
Last Update Posted: March 18, 2015
Last Verified: March 2015
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Sickle Cell Trait
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn